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lüll Substrate reduction therapy in mouse models of the glycosphingolipidoses Platt FM; Jeyakumar M; Andersson U; Heare T; Dwek RA; Butters TDPhilos Trans R Soc Lond B Biol Sci 2003[May]; 358 (1433): 947-54Substrate reduction therapy uses small molecules to slow the rate of glycolipid biosynthesis. One of these drugs, N-butyldeoxynojirimycin (NB-DNJ), shows efficacy in mouse models of Tay-Sachs, Sandhoff and Fabry diseases. This offers the prospect that NB-DNJ may be of therapeutic benefit, at least in the juvenile and adult onset variants of these disorders. The infantile onset variants will require an additional enzyme-augmenting modality if the pathology is to be significantly improved. A second drug, N-butyldeoxyglactonojirimycin, looks very promising for treating storage diseases with neurological involvement as high systemic dosing is achievable without any side-effects.|1-Deoxynojirimycin/analogs & derivatives/*pharmacology[MESH]|Animals[MESH]|Disease Models, Animal[MESH]|Enzyme Inhibitors/*pharmacology[MESH]|Mice[MESH]|Sphingolipidoses/*drug therapy/*metabolism[MESH]|Substrate Specificity[MESH] |